Don’t Weaken the F.D.A.’s Drug Approval Process

By Gregg Gonsalves, Mark Harrington and David A. Kessler

June 11, 2015

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CreditDavid Biskup

DURING the early days of the AIDS epidemic in the 1980s, there were no effective treatments against H.I.V., the virus that causes the disease. Because of this, many thousands of people died lingering deaths. The desperation of those times led to the rise of an activist movement that took to the streets and pressed government officials to expedite research on drugs to treat AIDS.

The danger of faster drug approval was that a devil’s bargain would be struck: quicker access to experimental drugs, but without first determining whether these drugs were safe and would improve health and extend life.

How that danger was navigated in the AIDS battle offers essential warnings for Congress as it considers the 21st Century Cures Act, which would lower standards for the approval of many medical products and potentially place patients at unnecessary risk.

As the AIDS crisis ballooned in the 1980s, the Food and Drug Administration became a target of groups like Act Up (AIDS Coalition to Unleash Power). Activists saw the agency, which approves prescription drugs, as a lumbering bureaucracy that delayed testing and early access to new drugs. It seemed indifferent to the sufferings of thousands, including some of the most vulnerable in our society: gay men, minorities, drug users and infants.

But as the scope of the AIDS crisis became apparent, the F.D.A. and its scientists began working closely with the AIDS community to speed access to experimental drugs. Novel processes were devised that expedited approvals of these new drugs by allowing their conditional release, pending further studies by the companies that developed them.

In the process, the agency transformed itself into a more flexible and creative institution that has introduced new ways to speed lifesaving treatments to patients. Crucially, these new procedures maintained the essential responsibility that drug companies have to patients and the American public: They would still have to show that the new drugs were safe and effective under the usual criteria required by the agency.

In turn, a new class of drugs — protease inhibitors — revolutionized treatment and transformed an epidemic that had eluded solutions. Patients rose from their deathbeds, reducing the death rate in the United States from AIDS to less than one-third of the 50,000 deaths a year in the mid-1990s.

This was made possible by robust research on the disease by the National Institutes of Health and the pharmaceutical industry, and by the F.D.A.’s Accelerated Approval Program, created in 1992.

It allowed for the preliminary approval of new treatments for serious or life-threatening diseases based on biomarkers that were reasonable proxies for showing that a treatment was working, rather than requiring outright proof that the drug could prevent illness and death. This allowed patients quick access to drugs, but required that manufacturers continued their research and proved with the usual set of clinical trials that their drugs actually benefited patients.

In the wake of the innovations of the early AIDS era, new-drug approvals by the F.D.A. have become the fastest in the world, and the agency has introduced new mechanisms such as fast-track and breakthrough drug designations that are increasingly being used to speed lifesaving new treatments to market.

But the 21st Century Cures Act could substantially lower the standards for approval of many medical products, potentially placing patients at unnecessary risk of injury or death. While the legislation does not mandate this approach, it opens the door to it.

For instance, the current version of the bill would allow consideration of drug approvals based on clinical experience, replacing scientific data from large numbers of patients in well-designed and controlled clinical trials.

Other provisions could mandate the use of biomarkers to approve a wider variety of drugs, far beyond just those for serious and life-threatening conditions.

Approval standards for medical devices, already lower than those for drugs, could become even less rigorous, allowing lifesaving devices like heart valves and stents to be revamped by manufacturers without the F.D.A. even reviewing the changes (though the agency would have to be notified).

Only a set of initial studies could be required for desperately needed new antibiotics. Worse, the critical, final studies to show that the new drugs save lives could be made optional. Though overuse of antibiotics has led to an epidemic of drug-resistant bacteria, the bill would give incentives to hospitals to prescribe these new antibiotics, encouraging more widespread use.

The F.D.A. has the expertise and legal and regulatory tools to expedite cures and better treatments. Congress should be wary of changing standards that paved the way for the development and approval of breakthrough drugs for AIDS and other diseases and that, importantly, have been proven to be effective and safe.

Gregg Gonsalves and Mark Harrington were members of Act Up/New York and co-founders of the Treatment Action Group. David A. Kessler was commissioner of the F.D.A. from 1990 to 1997.